Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that
researchers are scheduled to present data from clinical studies of
Soliris® (eculizumab) in patients with atypical hemolytic
uremic syndrome (aHUS), a genetic, life-long, ultra-rare disease
associated with vital organ failure and premature death. Data will be
presented at the annual meeting of the American Society of Nephrology
(ASN), being held from November 5 –10, 2013 in Atlanta, Georgia.
Presentations will include:
-
Results from a prospective clinical trial of Soliris in pediatric
patients with aHUS, following the published results of a retrospective
study in pediatric patients with aHUS
-
Results from an expanded adult population, representing the largest
prospective trial of Soliris in adult patients with aHUS
-
Three-year follow-up data from two pivotal Phase 2 extension studies
that highlight the long-term benefits of Soliris therapy in patients
with aHUS
Abstracts summarizing these presentations were published today on the
ASN website and can be accessed using the links below.
Soliris is approved in the United States, European Union, Japan and
other countries as a treatment for patients with aHUS. Soliris is also
approved in nearly 50 countries as a treatment for patients with
paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare
and life-threatening blood disorder characterized by complement-mediated
hemolysis (destruction of red blood cells). Both aHUS and PNH are
life-threatening, ultra-rare diseases that are caused by chronic
uncontrolled complement activation.
Soliris and aHUS
The following abstract will be presented in an oral session on Friday,
November 8, 2013 from 4:30 – 6:30 p.m. Eastern Standard Time (EST):
Abstract [FR-OR057]: “Eculizumab Inhibits Thrombotic Microangiopathy
(TMA) and Improves Renal Function in Adult Atypical Hemolytic Uremic
Syndrome (aHUS) Patients,” Fakhouri et al.
Accessible at: http://www.abstracts2view.com/asn_2013/view.php?nu=5593&terms=&type=abstract
The following abstracts will be presented in a poster session on
Saturday, November 9, 2013 from 10:00 a.m. – 12:00 p.m., Eastern
Standard Time (EST):
Abstract [SA-PO852]: “Eculizumab Maintains Efficacy in Atypical
Hemolytic Uremic Syndrome (aHUS) Patients with Progressing Thrombotic
Microangiopathy (TMA): 3-Year Update,” Gaber, et al.
Accessible at: http://www.abstracts2view.com/asn_2013/view.php?nu=5311&terms=&type=abstract
Abstract [SA-PO850]: “Eculizumab in Atypical Hemolytic Uremic Syndrome
(aHUS) Patients with Long Disease Duration and Chronic Kidney Disease
(CKD): Sustained Efficacy at 3 Years,” Delmas, et al.
Accessible at: http://www.abstracts2view.com/asn_2013/view.php?nu=5506&terms=&type=abstract
Abstract [SA-PO849]: “Eculizumab Inhibits Thrombotic Microangiopathy
(TMA) and Improves Renal Function in Pediatric Atypical Hemolytic Uremic
Syndrome (aHUS) Patients,” Greenbaum, et al.
Accessible at: http://www.abstracts2view.com/asn_2013/view.php?nu=5579&terms=&type=abstract
Abstract [SA-PO853]: “An Observational, Non-Interventional, Multicenter,
Multinational Registry of Patients with Atypical Hemolytic Uremic
Syndrome (aHUS): Initial Patient Characteristics,” Licht, et al.
Accessible at: http://www.abstracts2view.com/asn_2013/view.php?nu=5184&terms=&type=abstract
About Soliris
Soliris is a first-in-class terminal complement inhibitor developed from
the laboratory through regulatory approval and commercialization by
Alexion. Soliris is also approved in the US, European Union, Japan and
other countries as the first and only treatment for patients with
paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare
and life-threatening blood disorder, characterized by
complement-mediated hemolysis (destruction of red blood cells).
Soliris is also approved in the US, the European Union, Japan and other
countries as the first and only treatment for patients with atypical
hemolytic uremic syndrome (aHUS) to inhibit complement-mediated
thrombotic microangiopathy, a debilitating, ultra-rare and
life-threatening genetic disorder characterized by complement-mediated
thrombotic microangiopathy (blood clots in small vessels). The
effectiveness of Soliris in aHUS is based on the effects on thrombotic
microangiopathy (TMA) and renal function. Prospective clinical trials in
additional patients are ongoing to confirm the benefit of Soliris in
patients with aHUS. Soliris is not indicated for the treatment of
patients with Shiga-toxin E. coli related hemolytic uremic
syndrome (STEC-HUS).
Alexion's breakthrough approach in complement inhibition has received
the pharmaceutical industry's highest honors: the 2008 Prix Galien USA
Award for Best Biotechnology Product with broad implications for future
biomedical research and the 2009 Prix Galien France Award in the
category of Drugs for Rare Diseases.
Important Safety Information
The US product label for Soliris includes a boxed warning:
"Life-threatening and fatal meningococcal infections have occurred in
patients treated with Soliris. Meningococcal infection may become
rapidly life-threatening or fatal if not recognized and treated early.
Comply with the most current Advisory Committee on Immunization
Practices (ACIP) recommendations for meningococcal vaccination in
patients with complement deficiencies. Immunize patients with a
meningococcal vaccine at least 2 weeks prior to administering the first
dose of Soliris, unless the risks of delaying Soliris therapy outweigh
the risk of developing a meningococcal infection. (See Serious
Meningococcal Infections (5.1) for additional guidance on the management
of meningococcal infection.) Monitor patients for early signs of
meningococcal infections and evaluate immediately if infection is
suspected. Soliris is available only through a restricted program under
a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris
REMS, prescribers must enroll in the program. Enrollment in the Soliris
REMS program and additional information are available by telephone:
1-888-soliris (1-888-765-4747)."
In patients with PNH, the most frequently reported adverse events
observed with Soliris treatment in clinical studies were headache,
nasopharyngitis (runny nose), back pain and nausea. Soliris treatment of
patients with PNH should not alter anticoagulant management because the
effect of withdrawal of anticoagulant therapy during Soliris treatment
has not been established. In patients with aHUS, the most frequently
reported adverse events observed with Soliris treatment in clinical
studies were hypertension, upper respiratory tract infection, diarrhea,
headache, anemia, vomiting, nausea, urinary tract infection, and
leukopenia. Please see full prescribing information for Soliris,
including boxed WARNING regarding risk of serious meningococcal
infection.
About Alexion
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on
serving patients with severe and ultra-rare disorders through the
innovation, development and commercialization of life-transforming
therapeutic products. Alexion is the global leader in complement
inhibition, and has developed and markets Soliris®
(eculizumab) as a treatment for patients with PNH and aHUS, two
debilitating, ultra-rare and life-threatening disorders caused by
chronic uncontrolled complement activation. Soliris is currently
approved in nearly 50 countries for the treatment of PNH, and in the
United States, Europe, Japan and other countries for the treatment of
aHUS. Alexion is evaluating other potential indications for Soliris and
is pursuing development of four other innovative biotechnology product
candidates which are being investigated across additional severe and
ultra-rare disorders beyond PNH and aHUS. This press release and further
information about Alexion Pharmaceuticals, Inc. can be found at
www.alexionpharma.com.
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Copyright Business Wire 2013